BR112015008399A2 - composições para modular a expressão de c90rf72 - Google Patents

composições para modular a expressão de c90rf72

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Publication number
BR112015008399A2
BR112015008399A2 BR112015008399A BR112015008399A BR112015008399A2 BR 112015008399 A2 BR112015008399 A2 BR 112015008399A2 BR 112015008399 A BR112015008399 A BR 112015008399A BR 112015008399 A BR112015008399 A BR 112015008399A BR 112015008399 A2 BR112015008399 A2 BR 112015008399A2
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BR
Brazil
Prior art keywords
c9orf72
compositions
expression
c90rf72
modulating
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BR112015008399A
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English (en)
Other versions
BR112015008399A8 (pt
Inventor
E Swayze Eric
C Bennett Frank
Rigo Frank
M Freier Susan
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Isis Pharmaceuticals Inc
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Application filed by Isis Pharmaceuticals Inc filed Critical Isis Pharmaceuticals Inc
Publication of BR112015008399A2 publication Critical patent/BR112015008399A2/pt
Publication of BR112015008399A8 publication Critical patent/BR112015008399A8/pt

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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
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Abstract

resumo patente de invenção: "composições para modular a expressão de c90rf72". são aqui descritos métodos e composições para a redução da expressão de mrna c9orf72 e proteína em um animal com inibidores específicos c9orf72. tais métodos são úteis para tratar, prevenir ou melhorar doenças neurodegenerativas em um indivíduo com necessidade do mesmo. tais inibidores específicos de c9orf72 incluem compostos antissenso. exemplos de doenças neurodegenerativas que podem ser tratadas, prevenidas, e melhoradas com a administração de inibidores específicos c9orf72 incluem a esclerose lateral amiotrófica (als), demência fronto-temporal (ftd), síndrome de degeneração corticalbasal (cdb), síndrome parkinsoniana atípica, e degeneração olivopontocerellar (opcd).
BR112015008399A 2012-10-15 2013-10-15 Composto para modular a expressão de c90rf72, seu uso, oligonucleotídeo modificado, composto de fita dupla e composição BR112015008399A8 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261714132P 2012-10-15 2012-10-15
PCT/US2013/065073 WO2014062691A2 (en) 2012-10-15 2013-10-15 Compositions for modulating c9orf72 expression

Publications (2)

Publication Number Publication Date
BR112015008399A2 true BR112015008399A2 (pt) 2017-08-08
BR112015008399A8 BR112015008399A8 (pt) 2017-10-03

Family

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Application Number Title Priority Date Filing Date
BR112015008399A BR112015008399A8 (pt) 2012-10-15 2013-10-15 Composto para modular a expressão de c90rf72, seu uso, oligonucleotídeo modificado, composto de fita dupla e composição

Country Status (13)

Country Link
US (2) US10443052B2 (pt)
EP (1) EP2906258A4 (pt)
JP (3) JP6570447B2 (pt)
KR (1) KR20150070278A (pt)
CN (2) CN104968783B (pt)
AU (2) AU2013331434B2 (pt)
BR (1) BR112015008399A8 (pt)
CA (1) CA2888486A1 (pt)
HK (1) HK1212597A1 (pt)
IL (1) IL238266A0 (pt)
MX (2) MX370924B (pt)
RU (2) RU2020127664A (pt)
WO (1) WO2014062691A2 (pt)

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